Actively Recruiting
An Open, Single-Arm, Multi-Center Clinical Study to Evaluate the Safety and Tolerability of Regulatory T Cells (Tregs) In the Treatment of Neurodegenerative Diseases
Led by Novabio Therapeutics · Updated on 2025-09-25
12
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
N
Novabio Therapeutics
Lead Sponsor
T
The First Affiliated Hospital of Zhengzhou University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting an open-label, multi-center phase 1 clinical trial to assess the safety and tolerability of an investigational treatment using autologous human polyclonal regulatory T cells (NP001 cell injection) in patients with neurodegenerative diseases, specifically amyotrophic lateral sclerosis (ALS). The study aims to evaluate safety outcomes and identify the maximum tolerated dose. This study is sponsored by Novabio Therapeutics and focuses on patients diagnosed by established ALS criteria. Participants will undergo an apheresis procedure to collect their cells, which are then processed outside the body to enrich regulatory T cells. The investigational NP001 cell injections will be given intrathecally at doses of 1x10^6, 1x10^7, and 1x10^8 cells, administered up to three times at four-week intervals on Days 1, 29, and 57. Following the treatment period, participants will be monitored for approximately 12 months to assess safety and efficacy. During the study, participants will be regularly evaluated for treatment-related adverse events and dosage tolerance over six months. Functional assessments using the ALS Functional Rating Scale-Revised and Rasch Overall ALS Disability Scale will be conducted over the course of one year. The study involves various clinical and laboratory tests to monitor health status, and participants will be followed closely to capture both safety and functional outcomes throughout the trial duration.
CONDITIONS
Brief Title
Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients aged 18 to 70 years
- Diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS) meeting Gold Coast Diagnostic Criteria
- Stable dose of ALS treatment (riluzole and/or edaravone) for more than one month prior to study entry
- At least two weeks since major surgery or participation in other research trials
- Recovery from clinical toxicity with toxicity values resolved to less than grade 2
- Serum creatinine less than or equal to 2.0 mg/dL
- AST and ALT less than three times the upper limit of normal
- Bilirubin less than 1.5 except in Gilbert's disease
- Lung slow vital capacity greater than 70% of predicted normal
- No history of abnormal bleeding tendency
- Informed consent obtained prior to study procedures
You will not qualify if you...
- Uncontrolled infection
- Hypertension not controlled by three or more drugs
- Pulmonary embolism within 6 months prior to enrollment
- Significant heart conditions such as recent myocardial infarction, severe heart failure, uncontrolled angina, or severe arrhythmias
- History of coronary artery bypass grafting or angioplasty without cardiology approval
- Positive for HIV, hepatitis B or C
- Pregnant or lactating patients
- Unwillingness to use contraception if of childbearing potential
- Participation in other interventional studies
- Treatment with investigational drug, biologic, or device within 30 days or five half-lives prior to screening
- Prior ALS gene or cell therapy
- Significant tumor, liver, kidney, or other uncontrolled diseases
- Presence of feeding tube
- Current use of certain antipsychotic, antiepileptic, or antiarrhythmic drugs
- Significant risk of suicide as determined by investigator
- Other conditions deemed unsuitable by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 8 weeks
Participants receive up to three intrathecal injections of NP001 cell therapy, each separated by 4 weeks.
3 dosing visits
Duration - Up to 12 months
Participants are monitored for safety and efficacy parameters after the final treatment.
Regular follow-up visits over 12 months
Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, China, 450052
Actively Recruiting
Research Team
M
Mingqi Lu, MD., PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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